From rare to revolutionary: how science can reach every patient

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Re:State were delighted to host a high-level roundtable exploring the gap between scientific progress and service delivery for patients with rare diseases. Around 3.5 million people in the UK live with a rare disease, yet fewer than 5 per cent currently have approved treatments, and many patients require complex, lifelong care.

Despite significant scientific advances such as novel gene therapies and improved diagnostics, the NHS continues to struggle to deliver access, coordination and scale. Fragmented data, delays in approvals and uncertainty around reimbursement all limit how quickly innovation reaches patients.

The discussion highlighted the coordination of care as a central, often missing link in the patient journey, alongside the case for grouping rare diseases by shared characteristics to make research and service delivery easier to organise at scale.

Re:State were honoured to be joined by a diverse group of industry and public sector leaders, with the discussion introduced by Louise Fish, Chair of the Rare Diseases Advisory Group, NHS England; Colin Wilson, Deputy Director of Research Infrastructure, Office for Life Sciences, Department of Health and Social Care; and Lesley Perkin, Patient Engagement & Policy Lead for Rare Diseases and Epilepsy, UCB, UK&I. This write-up brings together the key themes which emerged during the discussion.

This roundtable and write-up were kindly supported by UCB.